NZ600616A - Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases - Google Patents
Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseasesInfo
- Publication number
- NZ600616A NZ600616A NZ600616A NZ60061610A NZ600616A NZ 600616 A NZ600616 A NZ 600616A NZ 600616 A NZ600616 A NZ 600616A NZ 60061610 A NZ60061610 A NZ 60061610A NZ 600616 A NZ600616 A NZ 600616A
- Authority
- NZ
- New Zealand
- Prior art keywords
- mrna
- protein
- lipids
- liposome
- enzyme
- Prior art date
Links
- 108020004999 messenger RNA Proteins 0.000 title abstract 7
- 102000004169 proteins and genes Human genes 0.000 title abstract 7
- 108090000623 proteins and genes Proteins 0.000 title abstract 7
- 102000004190 Enzymes Human genes 0.000 title abstract 4
- 108090000790 Enzymes Proteins 0.000 title abstract 4
- 208000026350 Inborn Genetic disease Diseases 0.000 title 1
- 230000003416 augmentation Effects 0.000 title 1
- 208000016361 genetic disease Diseases 0.000 title 1
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 abstract 4
- 150000002632 lipids Chemical class 0.000 abstract 4
- 239000002502 liposome Substances 0.000 abstract 4
- 125000002091 cationic group Chemical group 0.000 abstract 3
- 235000012000 cholesterol Nutrition 0.000 abstract 2
- 239000003814 drug Substances 0.000 abstract 2
- 210000004185 liver Anatomy 0.000 abstract 2
- 238000004519 manufacturing process Methods 0.000 abstract 2
- 230000002035 prolonged effect Effects 0.000 abstract 2
- 230000010473 stable expression Effects 0.000 abstract 2
- 230000007812 deficiency Effects 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
- A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
- C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Dispersion Chemistry (AREA)
- Biophysics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Dermatology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Steroid Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| NZ700688A NZ700688A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26565309P | 2009-12-01 | 2009-12-01 | |
| PCT/US2010/058457 WO2011068810A1 (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ600616A true NZ600616A (en) | 2014-11-28 |
Family
ID=44115246
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ600616A NZ600616A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ750556A NZ750556A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ733634A NZ733634A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ700688A NZ700688A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ716192A NZ716192A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ750556A NZ750556A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ733634A NZ733634A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ700688A NZ700688A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| NZ716192A NZ716192A (en) | 2009-12-01 | 2010-11-30 | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
Country Status (19)
| Country | Link |
|---|---|
| US (9) | US20110244026A1 (lt) |
| EP (5) | EP3338765B1 (lt) |
| AU (1) | AU2010326132B9 (lt) |
| CA (2) | CA3077990A1 (lt) |
| CY (2) | CY1120272T1 (lt) |
| DK (2) | DK3338765T3 (lt) |
| ES (3) | ES2666559T3 (lt) |
| HR (2) | HRP20180696T1 (lt) |
| HU (2) | HUE038039T2 (lt) |
| LT (2) | LT2506857T (lt) |
| ME (2) | ME03327B (lt) |
| NO (1) | NO2506857T3 (lt) |
| NZ (5) | NZ600616A (lt) |
| PL (2) | PL2506857T3 (lt) |
| PT (2) | PT2506857T (lt) |
| RS (2) | RS57314B1 (lt) |
| SI (2) | SI3338765T1 (lt) |
| TR (1) | TR201901311T4 (lt) |
| WO (1) | WO2011068810A1 (lt) |
Families Citing this family (262)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101734955B1 (ko) | 2008-11-07 | 2017-05-12 | 메사추세츠 인스티튜트 오브 테크놀로지 | 아미노알콜 리피도이드 및 그의 용도 |
| PT2506857T (pt) | 2009-12-01 | 2018-05-14 | Translate Bio Inc | Entrega de arnm para o acréscimo de proteínas e enzimas em doenças genéticas humanas |
| CA2804492A1 (en) | 2010-07-06 | 2012-01-12 | Novartis Ag | Immunisation of large mammals with low doses of rna |
| HUE047796T2 (hu) | 2010-07-06 | 2020-05-28 | Glaxosmithkline Biologicals Sa | RNS bevitele több immunútvonal bekapcsolására |
| US9770463B2 (en) | 2010-07-06 | 2017-09-26 | Glaxosmithkline Biologicals Sa | Delivery of RNA to different cell types |
| US11291635B2 (en) | 2010-07-06 | 2022-04-05 | Glaxosmithkline Biological Sa | Virion-like delivery particles for self-replicating RNA molecules |
| WO2012006378A1 (en) | 2010-07-06 | 2012-01-12 | Novartis Ag | Liposomes with lipids having an advantageous pka- value for rna delivery |
| CA2807552A1 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| WO2012027675A2 (en) | 2010-08-26 | 2012-03-01 | Massachusetts Institute Of Technology | Poly(beta-amino alcohols), their preparation, and uses thereof |
| LT4066819T (lt) | 2010-08-31 | 2023-04-11 | Glaxosmithkline Biologicals Sa | Mažos liposomos, skirtos imunogeną koduojančios rnr pristatymui |
| PT4066856T (pt) | 2010-08-31 | 2023-01-02 | Glaxosmithkline Biologicals Sa | Lipossomas peguilados para entrega de arn codificador de imunogénio |
| EP2614680B1 (de) * | 2010-09-09 | 2017-06-28 | Saint-Gobain Glass France | Transparente scheibe mit heizbeschichtung |
| CN103429606A (zh) | 2010-10-01 | 2013-12-04 | 现代治疗公司 | 设计核酸及其使用方法 |
| CN103269713B (zh) | 2010-10-11 | 2016-01-20 | 诺华有限公司 | 抗原递送平台 |
| WO2012075040A2 (en) | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| WO2012135025A2 (en) | 2011-03-28 | 2012-10-04 | Massachusetts Institute Of Technology | Conjugated lipomers and uses thereof |
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